Acute LeukemiaPhase 1/2NCT05589896
A First-in-Human Study of HLA-Partially to Fully Matched Allogenic Cryopreserved Deceased Donor Bone Marrow Transplantation for Patients With Hematologic Malignancies
A Phase 1/2 study testing Ossium HPC Marrow, Bone Marrow Transplant, Pre-transplant conditioning - Myeloablative (MAC) and Pre-transplant conditioning - Reduced Intensity (RIC) in people with Acute Leukemia and Acute Lymphoblastic Leukemia, run by Ossium Health, Inc. at 9 sites in United States.
Trial summary
The goal of this clinical trial is to determine the safety and feasibility of allogeneic transplantation with bone marrow from a deceased donor in patients with acute and chronic leukemias, myelodysplastic syndrome, and certain lymphomas. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- What is being tested?
- Ossium HPC Marrow, Bone Marrow Transplant, Pre-transplant conditioning - Myeloablative (MAC) and Pre-transplant conditioning - Reduced Intensity (RIC)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 9 sitesUnited States
- Study dates
- 16 Aug 2024to 31 May 2029
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient has the ability to provide informed consent according to the applicable regulatory and local institutional requirements
- Male or female, aged ≥18 and ≤65 years for patients receiving MAC (Regimen A or Regimen B); aged ≥18 and ≤75 years for patients receiving RIC (Regimen C or D)
- Patient must require allogeneic HCT per the discretion of the treating physician
- Patient must be high-resolution, HLA partially or fully matched (4-8/8 allele matched at HLA-A, -B, -C, DRB1) to an available Ossium HPC, Marrow product
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Diagnosed with malignant hematologic disease including:
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- Acute leukemia [acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), acute biophenotypic leukemia (ABL), or acute undifferentiated leukemia (AUL)], MDS without fibrosis, or chronic leukemia (CLL, CML) in the first remission or beyond with ≤5% marrow blasts documented by bone marrow assessment and no circulating blasts or extra-medullary disease within 42 days prior to anticipated start of conditioning
- Chemosensitive non-Hodgkin's lymphomas, Hodgkin's lymphoma, or cutaneous T cell lymphomas in the first remission or beyond documented by PET/CT imaging and bone marrow assessment within 42 days prior to anticipated start of conditioning
- Karnofsky performance status score ≥70% (MAC) or ≥60% (RIC)
- HCT comorbidity index (HCT-CI) ≤5
- Adequate organ function defined as:
- Cardiac: LVEF at rest ≥40% (RIC) or LVEF at rest ≥45% (MAC)
- Pulmonary: DLCO, FEV1, FVC ≥50% predicted by pulmonary function tests (PFTs). DLCO value may be corrected for hemoglobin.
- Hepatic: total bilirubin ≤2.0 mg/dL, and ALT, AST, and ALP <3 x upper limit normal (ULN), unless ALT, AST, and/or ALP are disease related
- Renal: SCr within 1.5x normal range for age. If SCr is outside normal range for age, CrCl> 60 mL/min/1.73m2 must be obtained (measured by 24-hour urine specimen or nuclear glomerular filtration rate (GFR), or calculated GFR)
Who cannot join
- Availability of suitable graft from living donor (defined as 7/8 or 8/8 HLA-matched related or unrelated donors, haploidentical donors, or cord blood donors)
- Prior autologous or allogeneic HCT
- Pregnancy or lactation
- Ongoing treatment with an investigational drug used for disease-related treatment within 5 half-lives of the drug
- Current uncontrolled bacterial, viral or fungal infection defined as currently taking medication with evidence of progression of clinical symptoms or radiologic findings
- Any condition(s) or diagnosis, both physical or psychological, or physical exam finding that in the investigator's opinion precludes participation
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Emory University - Winship Cancer Institute
Atlanta, Georgia, 30322
St. David's South Austin Medical Center
Austin, Texas, 78745
Henry Ford Cancer Institute
Detroit, Michigan, 48202
City of Hope
Duarte, California, 91010
TriStar Bone Marrow Transplant
Nashville, Tennessee, 37203
Oregon Health and Science University
Portland, Oregon, 97239
University of Utah - Huntsman Cancer Institute
Salt Lake City, Utah, 84112
Methodist Hospital, Texas Transplant
San Antonio, Texas, 78229
Moffitt Cancer Center
Tampa, Florida, 33612
About this study
The goal of this clinical trial is to determine the safety and feasibility of allogeneic transplantation with bone marrow from a deceased donor in patients with acute and chronic leukemias, myelodysplastic syndrome, and certain lymphomas. Patients will either receive myeloablative conditioning or reduced intensity conditioning regimen prior to the transplant. Patients will be followed for 56 days for safety endpoints and remain in follow-up for one year.
- Conditions
- Acute Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Biphenotypic Leukemia, Acute Undifferentiated Leukemia, CLL (Chronic Lymphocytic Leukemia)
- Being tested
- Ossium HPC Marrow, Bone Marrow Transplant, Pre-transplant conditioning - Myeloablative (MAC), Pre-transplant conditioning - Reduced Intensity (RIC), Post-transplant treatment
- Keywords
- Leukemia, Hematologic Diseases, ALL, AML, ABL, AUL, Bone Marrow Transplant, Lymphoma, MDS, CLL, CML
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Leukemia, Biphenotypic, Acute, Leukemia, B-Cell, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Anemia, Refractory, with Excess of Blasts, Hodgkin Disease, Lymphoma, T-Cell, Cutaneous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ossium HPC Marrow, Bone Marrow TransplantOther
Hematopoetic Cell Transplantation
Pre-transplant conditioning - Myeloablative (MAC)Other
Also called Busuflex, Fludara, TBI
Regimen A or Regimen B
Pre-transplant conditioning - Reduced Intensity (RIC)Other
Also called Fludara, Cytoxan, Mesnex, TBI
Regimen C or Regimen D
Post-transplant treatmentOther
Also called Cytoxan, Mesnex, CellCept, Filgrastim
Post-transplant treatment
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Bone Marrow Transplant with Ossium HPC, Marrow
Pre-transplant myeloablative conditioning treatment with:
Regimen A(MAC): Busulfan and Fludarabine
[OR]
Regimen B(MAC): Fludarabine and Total Body Irradiation
[OR]
Regimen C(RIC): Fludarabine, Cyclophosphamide, and Total Body Irradiation
[OR]
Regimen D (RIC): Fludarabine, Melphalan, and Total Body Irradiation
Post-Transplant treatment with Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, and Filgrastim Receives: Other: Ossium HPC Marrow, Bone Marrow Transplant; Other: Pre-transplant conditioning - Myeloablative (MAC); Other: Pre-transplant conditioning - Reduced Intensity (RIC); Other: Post-transplant treatment |
| Cohort 2 | Experimental | *Open to enrollment after DSMB review of Cohort 1 safety events through Day 56*
Bone Marrow Transplant with Ossium HPC, Marrow
Pre-transplant conditioning treatment with:
Regimen A(MAC): Busulfan and Fludarabine
[OR]
Regimen B(MAC): Fludarabine and Total Body Irradiation
[OR]
Regimen C(RIC): Fludarabine, Cyclophosphamide, and Total Body Irradiation
[OR]
Regimen D(RIC): Fludarabine, Melphalan, and Total Body Irradiation
Post-Transplant treatment with Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, and Filgrastim Receives: Other: Ossium HPC Marrow, Bone Marrow Transplant; Other: Pre-transplant conditioning - Myeloablative (MAC); Other: Pre-transplant conditioning - Reduced Intensity (RIC); Other: Post-transplant treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Neutrophil Engraftment
Neutrophil engraftment is defined as achieving an absolute neutrophil count (ANC) of greater than or equal to 500/µL for 3 consecutive measurements on 3 different days by Day 28.
Measured: Day 28
Serious Adverse Events
Occurrence of any event classified as SAE. The time of occurrence of each serious adverse event will be recorded.
Measured: Day 56
CTCAE Grade 3/4 Adverse Events (AEs)
Occurrence of any event classified as grade 3/4 AE attributed to Ossium HPC, Marrow per the CTCAE v5.0 guidelines. The time of the occurrence of each event will be recorded.
Measured: Day 56
CTCAE Grade 3/4 Adverse Events (AEs) attributed to infusion of Ossium HPC, Marrow
Occurrence of any event classified as grade 3 or higher attributed to Ossium HPC, Marrow infusion per the CTCAE v5.0 guidelines. The time of the occurrence will be recorded.
Measured: Day 28
Death
The time of death will be recorded for each expired patient.
Measured: Day 56
Secondary outcomes (7)
Cumulative incidences of neutrophil engraftment
Neutrophil engraftment in defined as achieving an absolute neutrophil count (ANC) of greater than or equal to 500/µL for 3 consecutive measurements on different days by Day 28.
Measured: Day 28
Cumulative incidences of platelet recovery
Platelet recovery is defined as platelets greater than or equal to 20,000/µL for 3 consecutive days in the absence of transfusion for 7 consecutive days by Day 56.
Measured: Day 56
Cumulative incidence of disease relapses
The cumulative incidence of relapse is measured from the date of transplant (Day 0) until the date of relapse or progression; patients not known to have relapsed are censored on the date they were last examined; patients who died without relapse are counted as a competing cause of failure.
Measured: Day 365
Transplant-related mortality (TRM)
TRM is defined as death without evidence of disease progression or recurrence.
Measured: Day 100 and Day 365
Cumulative incidences of acute (aGVHD) Graft Versus Host Disease
aGVHD is defined as any skin, gastrointestinal or liver abnormalities fulfilling the criteria of grades II-IV or grades III-IV.
Measured: Day 100, Day 180, and Day 365
Cumulative incidences of chronic (cGVHD) Graft Versus Host Disease
cGVHD is defined per National Institutes of Health (NIH) Consensus Criteria and includes organ involvement and severity, and overall global composite score (mild/moderate/severe).
Measured: Day 100, Day 180, and Day 365
Incidence of clinically-significant infections
A clinically significant infection is defined as any microbiologic or radiographic infection for which antimicrobial therapy was administered.
Measured: Day 100 and Day 365
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2022
- Study start16 Aug 2024
- Primary completion14 Jul 2026
- Study completion31 May 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- All patients will receive Ossium HPC, Marrow product. Study arms will be based on order of enrollment and planned conditioning regimen.
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ossium Health, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05589896
- Sponsor's protocol ID
- PRESERVE I
- Other IDs
- 75A50124C00032
- Status last verified
- Sep 2026
- First submitted
- 18 Oct 2022
- Last updated
- 10 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No